Versartis Trial in Pre-pubertal Japanese Children With Growth Hormone Deficiency (GHD) to Assess Long-Acting Growth Hormone (Somavaratan, VRS-317)

A Randomized, Phase 2/3, Open-Label, Multi-center Study of the Pharmacokinetics, Pharmacodynamics, Safety and Efficacy of A Long-acting Human Growth Hormone (Somavaratan, VRS-317) in Pre-pubertal Japanese Children With Growth Hormone Deficiency (GHD)

The trial will consist of three stages: 1) a 30 day Phase 2 PK and PD evaluation of somavaratan, 2) an optional Phase 2 Extension and 3) a 12 month Phase 3 safety and efficacy stage. Upon completion of the PK/PD stage, the PK/PD profiles for the GHD children in this study will be compared to the PK/PD profiles for the GHD children treated in the Western study Phase 1b/2a study (Protocol 12VR2) and identify the somavaratan dose to be used in the Phase 3 stage in Japan. The Phase 3 stage will continue dosing for 12 months to obtain safety and efficacy data on 48 subjects.

Study Overview

• Status: Terminated
• Conditions: Growth Disorders; Pediatric Growth Hormone Deficiency
• Intervention / Treatment: Drug: Somavaratan (VRS-317)

Detailed Description

The trial will consist of three stages: 1) a 30 day Phase 2 PK and PD evaluation of somavaratan, 2) an optional Phase 2 Extension and 3) a 12 month Phase 3 safety and efficacy stage. The study is a randomized, multi-center, open label study. The primary endpoint is height velocity at 12 months.

• Study Type: Interventional
• Enrollment (Actual): 41
• Phase: Phase 2; Phase 3

Contacts and Locations

Study Locations

• Japan
  • Sapporo, Japan
    • Hokkaido University Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 5 months to 6 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Chronological Age ≥ 3.0 years and ≤ 9.0 years (girls) or ≤ 10.0 years (boys)
• Pre-pubertal status
• Diagnosis of GHD as documented by two or more GH stimulation test results
• Height SD score ≤ -2.0 at screening
• Weight for Stature ≥ 10th percentile
• IGF-I SD score ≤ -1.0 at screening
• Delayed bone age

Exclusion Criteria:

• Prior treatment with any growth promoting agent
• History of, or current, significant disease
• Chromosomal aneuploidy, significant gene mutations (other than those that cause GHD) or confirmed diagnosis of a named syndrome
• Birth weight and/or birth length less than 5th percentile for gestational age
• A diagnosis of Attention Deficit Hyperactivity Disorder
• Daily use of anti-inflammatory doses of glucocorticoid
• Prior history of leukemia, lymphoma, sarcoma or cancer
• Ocular findings suggestive of increased intracranial pressure and/or retinopathy at screening
• Significant spinal abnormalities including scoliosis, kyphosis and spina bifida variants
• Significant abnormality in screening laboratory studies

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Phase 2: Somavaratan (VRS-317); Active treatment arm	Drug: Somavaratan (VRS-317); Long acting recombinant human growth hormone; Other Names: Long acting recombinant human growth hormone
Experimental: Phase 3: Somavaratan (VRS-317); Somavaratan long acting recombinant human growth hormone administered subcutaneously twice-monthly	Drug: Somavaratan (VRS-317); Long acting recombinant human growth hormone; Other Names: Long acting recombinant human growth hormone

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Efficacy (Annual Height velocity)	Annual Height velocity.	12 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Pharmacodynamics (IGF-I responses to study drug administration)	IGF-I responses to study drug administration.	12 months
Pharmacodynamics (IGFBP-3 responses to study drug administration)	IGFBP-3 responses to study drug administration.	12 months
Safety (Number of subjects with adverse events )	Number of subjects with adverse events (including repeat dose immunogenicity).	12 months
Safety (Concomitant medications)	Concomitant medications	12 months
Safety (Safety labs)	Safety labs	12 months
Safety (Vital signs)	Vital signs	12 months
Safety (Physical Exams)	Physical Exams	12 months

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Secondary Efficacy (Change in height SDS)	Change in height SDS.	12 months
Secondary Efficacy	Change in body weight	12 months
Secondary Efficacy	Change in body mass index.	12 months
Secondary Efficacy (Change in bone age)	Change in bone age.	12 months
Secondary Efficacy (Change in pubertal staging.)	Change in pubertal staging.	12 months

Collaborators and Investigators

• Sponsor: Versartis Inc.
• Investigators: Study Director: Will Charlton, MD, Vesrartis

Study record dates

Study Major Dates

• Study Start (Actual): August 8, 2015
• Primary Completion (Actual): November 30, 2017
• Study Completion (Actual): November 30, 2017

Study Registration Dates

• First Submitted: March 23, 2015
• First Submitted That Met QC Criteria: April 8, 2015
• First Posted (Estimate): April 9, 2015

Study Record Updates

• Last Update Posted (Actual): July 25, 2022
• Last Update Submitted That Met QC Criteria: July 22, 2022
• Last Verified: July 1, 2022

More Information

Terms related to this study

• Keywords: GHD
• Additional Relevant MeSH Terms: Pathologic Processes; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Musculoskeletal Diseases; Hypothalamic Diseases; Bone Diseases; Bone Diseases, Endocrine; Pituitary Diseases; Dwarfism; Bone Diseases, Developmental; Hypopituitarism; Dwarfism, Pituitary; Endocrine System Diseases; Growth Disorders; Physiological Effects of Drugs; Hormones, Hormone Substitutes, and Hormone Antagonists; Hormones
• Other Study ID Numbers: J14VR5

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No